Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Heterocyclic carbon compounds containing a hetero ring...
Patent
1996-04-03
1998-05-19
Henley, III, Raymond
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Heterocyclic carbon compounds containing a hetero ring...
A61K 31495
Patent
active
057536562
DESCRIPTION:
BRIEF SUMMARY
This application is a 371 of international application PCT/JP95/01550 filed Aug. 4, 1995
BACKGROUND OF THE INVENTION
1. Field of Application in Industry
The present invention relates to an agent for treating spinocerebellar degeneration containing as an effective ingredient a compound represented by the formula (I): ##STR2## represent a single bond while R.sup.3 represents --CH(OH)CH(OH)CH.sub.3, --CH(OCOCH.sub.3)CH(OCOCH.sub.3)CH.sub.3, --CH.sub.3, --CH.sub.2 OH or a phenyl group when R.sup.1 and R.sup.2 each represents a hydrogen atom, or --COCH(OH)CH.sub.3 when R.sup.1 and R.sup.2 together represent a single bond! or its pharmaceutically acceptable salt.
2. Prior Art
Spinocerebellar degeneration is a disease causing the degenerations of the nervous system mainly occurring in the cerebellar system, and can be roughly divided into non-hereditary and hereditary forms. The disease causes the systematic degeneration of the spinal cord, the cerebellum and its output and/or input pathway, and the cerebral basal ganglia with the subsequent attenuation of their neural functions. It is an intractable disease mainly characterized by the cerebellar ataxia, such as ataxic gait and tremor of the extremities, leading to manifestations of speech disturbance, dysphagia, abnormal ventilation and involuntary movements such as dystonia, thereby substantially interfering with activities of daily life. The cause of the disease remains unknown. With regard to its treatment, it has been reported that a marked improvement of neurologic symptoms was observed in a patient with Machado-Joseph disease, one of the spinocerebellar degeneration, following administration of a combined antimicrobial preparation of sulfamethoxazole-trimethoprim (hereinafter referred to as S-T preparation) (Archives of Neurology, 1988; 45: 210-213). Because of its serious side-effects, however, the drug has not been developed as therapeutic agent for administration to patients with this disease. Therefore, neither drugs for causal treatment nor those for symptomatic treatment of the disease which definitely provide an alleviation of symptoms, have as yet been established, and the advent of an effective therapeutic agent is keenly sought.
The effective ingredient, compound (I), of the therapeutic agent of the present invention is a known compound with proven clinical efficacy in the treatment of malignant hyperphenylalaninemia, depression, Parkinson disease, and other disorders. For example, refer to Japanese Patent Application Public Disclosure (KOKAI) Nos. 25323/84, 76086/84, 277618/86 and 267781/88.
SUMMARY OF THE INVENTION
The object of the present invention is to provide a safe therapeutic agent devoid of any side-effect, which reverses an attenuation of brain neural function with the consequent symptomatic amelioration, thus enabling the patient to lead a normal life.
In order to solve the above problem, the present inventors took note of the afore-mentioned improvement of neurologic symptoms observed following the administration of the antimicrobial agent S-T preparation in a patient with Machado-Joseph disease, one of the spinocerebellar degeneration (Archives of Neurology, 1988; 45: 210-213). Through an elaborate research into the mechanisms of pharmacologic actions of S-T preparation, the present inventors hypothesized that the drug produced improvement of neurologic symptoms by way of increasing the metabolic turnover rate in the brain tetrahydrobiopterin which is present in minute quantities in humans. In view of this, the inventors performed a clinical trial with tetrahydrobiopterin as the treatment of patients with Machado-Joseph disease. As a result, they discovered a remarkable amelioration of the neurologic symptoms to achieve the present invention. The present invention, relates to an agent for treating Machado-Joseph disease as well as other spinocerebellar degeneration having spinocerebellar lesion and pathology in common with Machado-Joseph disease.
DETAILED DESCRIPTION OF THE INVENTION
Therefore, the present invention is an a
REFERENCES:
patent: 4774244 (1988-09-01), Curtius et al.
patent: 4920122 (1990-04-01), Naruse et al.
J. Pharmacol. Exp. Ther., 242(2), pp. 514-522, 1987.
Research Report by Medical Application Research Foundation, 12, pp. 233-240, 1994.
Sakai, Tetsuo, Clinical Characteristics of Joseph Disease. A proposal of diagnostic criteria.; 1989, pp. 216-252.
Mello, Karen et al., Effects of Sulfamethoxazole and Trimethoprim on Neurologic Dysfunction in a Patient With Joseph's Disease, Arch. Neurol., vol. 45, Feb. 1988, pp. 210-213.
Antoku Yasunobu
Matsuishi Toyojiro
Sakai Tetsuo
Henley III Raymond
Suntory Limited
LandOfFree
Method for treating spinocerebellar degeneration does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Method for treating spinocerebellar degeneration, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Method for treating spinocerebellar degeneration will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1853238